Micro- and nanoscale delivery systems for macromolecules: gene delivery

大分子微米级和纳米级递送系统：基因递送

• 批准号: 155455-2008
• 负责人: Foldvari, Marianna
• 金额: $ 1.42万
• 依托单位: University of Waterloo
• 依托单位国家: 加拿大
• 项目类别: Discovery Grants Program - Individual
• 财政年份: 2011
• 资助国家: 加拿大
• 起止时间: 2011-01-01 至 2012-12-31
• 项目状态: 已结题
• 来源: https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=476063
• 关键词: Micro; nanoscale; delivery; systems; macromolecules

One of the biggest challenges in gene therapy -- inserting genes into an individual's cells and tissues to treat a disease -- is successfully delivering DNA into cells, followed by the DNA being expressed. In our search for suitable materials to package DNA, we have identified a group of compounds known as gemini surfactants that can condense, protect and transport DNA into cells. Using these electrically charged compounds, we have developed a non-viral nanoparticle-based technology to deliver genes through the skin. Non-viral delivery systems have significant advantages relative to viral systems, as they are safer, cheaper and easier to prepare. Their major disadvantage, however, is that they are not as efficient, a deficiency this proposed research will address.

基因治疗中最大的挑战之一--将基因植入个人的细胞和组织以治疗疾病--是成功地将DNA输送到细胞中，然后表达DNA。在寻找合适的材料包装DNA的过程中，我们已经确定了一组被称为双子表面活性剂的化合物，它们可以凝聚、保护DNA并将其输送到细胞中。利用这些带电化合物，我们开发了一种基于非病毒纳米颗粒的技术，可以通过皮肤传递基因。与病毒系统相比，非病毒递送系统具有显著的优势，因为它们更安全、更便宜、更容易制备。然而，它们的主要缺点是效率不高，这项拟议的研究将解决这一不足。